EMEA

先月、EMEAにオーファンドラッグ指定された医薬品のリストが発表されています。

Treatment of carcinoid tumours
(S)-ethyl 2-amino-3-(4-(2-amino-6-((R)-1-(4-chloro-2-(3-methyl-1H-pyrazol-1-yl)phenyl)-2,2,2-trifluoroethoxy)pyrimidin-4-yl)phenyl)propanoate

Treatment of acute myeloid leukaemia
26 base single stranded phosphodiester DNA oligonucleotide

Treatment of Duchenne muscular dystrophy
Adeno-associated viral vector containing modified U1 snRNA

Treatment of myelodysplastic syndromes
Allogeneic ex vivo expanded umbilical cord blood cells

Treatment of chronic myeloid leukaemia
Allogeneic ex vivo expanded umbilical cord blood cells

Treatment of familial adenomatous polyposis
Eicosapentaenoic acid

Treatment of anal fistula
Expanded human allogeneic mesenchymal adult stem cells extracted from adipose tissue

Treatment of angioedema caused by C1-inhibitor deficiency
Human C1-inhibitor

Prevention of graft rejection during pancreatic islet transplantation
Low molecular weight dextran sulphate

Treatment of acromegaly
Pasireotide

Treatment of Cushing’s disease
Pasireotide

Treatment of multiple myeloma
Pomalidomide

Treatment of Hodgkin lymphoma
Recombinant antibody construct against human CD30 and CD16A

Prevention of scarring post glaucoma filtration surgery
Recombinant human serum amyloid P

Treatment of haemophilia A
Sequence-modified recombinant human factor VIIa

Treatment of haemophilia B
Sequence-modified recombinant human factor VIIa

Treatment of acute lung injury
Human tumour necrosis factor alfa-derived peptide Cys-Gly-Gln-Arg-Glu-Thr-Pro-Glu-Gly-Ala-Glu-Ala-Lys-Pro-Trp-Tyr-Cys

Treatment of soft tissue sarcoma
(-)-trans-3-(5,6-dihydro-4H-pyrrolo[3,2,1-ij]quinolin-1yl)-4-(1H-indol-3-yl) pyrrolidine-2, 5-dione

詳しくはこちらをご覧ください。

http://ec.europa.eu/enterprise/pharmaceuticals/register/orphreg.htm

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